Hemophilia A Pipeline

DelveInsight’s, “Hemophilia A - Pipeline Insight, 2025” report provides comprehensive insights about 25+ companies and 30+ pipeline drugs in Hemophilia A pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

Geography Covered

• Global coverage

 

Hemophilia A: Understanding

Hemophilia A: Overview

Hemophilia represents a group of inherited bleeding disorders that disrupt the normal process of blood coagulation, making affected individuals prone to abnormal or excessive bleeding. Among these, classical hemophilia, commonly referred to as hemophilia A, is the most prevalent type. This condition arises from a congenital deficiency of clotting factor VIII, a protein essential for forming stable blood clots. As a result, patients often experience prolonged and excessive bleeding episodes, which may occur spontaneously without apparent cause or in response to even minor injuries, surgical procedures, or trauma. Over time, recurrent bleeding can also lead to complications such as joint damage and reduced quality of life, underscoring the clinical significance of early diagnosis and effective management.

Hemophilia A is typically confirmed after the onset of bleeding symptoms, which may arise spontaneously or following minor trauma. Key manifestations include spontaneous intracranial hemorrhage in newborns, excessive bleeding after circumcision, painful atraumatic hemarthrosis, and unexplained bruising during early mobility, and significant musculocutaneous bleeding either spontaneously or after intramuscular injections. In young children, frequent falls or minor impacts can lead to extensive bruising and soft tissue hemorrhage, sometimes resembling signs of child abuse.

It is classified by severity into severe, moderate, and mild forms, depending on residual factor VIII activity. Severe hemophilia A is often diagnosed in infancy and is marked by frequent spontaneous bleeding, including joint and muscle hemorrhages, with untreated patients experiencing two to five bleeding episodes monthly and risk of life-threatening intracranial bleeds. Moderate hemophilia A presents with less frequent spontaneous bleeding, usually triggered by minor trauma, with diagnosis often made before age six; untreated patients may have bleeding episodes ranging from once a month to once a year, resembling the manifestations of severe disease but with lower frequency. Mild hemophilia A is typically asymptomatic until surgery, dental extractions, or major injuries provoke bleeding, and diagnosis often occurs later in life; bleeding frequency varies widely, from a few times annually to once in a decade. Additionally, affected females with reduced factor VIII activity may experience bleeding comparable to males, while about a quarter of females with normal activity still show a bleeding tendency, likely due to impaired stress-induced factor VIII elevation.

When the vascular endothelium is injured, the hemostatic process activates the coagulation cascade to restore vessel integrity and prevent excessive bleeding. At the site of injury, platelets adhere and become activated, triggering the release of clotting factors and initiating fibrin formation, which together form a platelet–fibrin plug to arrest bleeding. Factor VIII, whose deficiency underlies hemophilia A, plays a critical role by amplifying thrombin generation and promoting robust fibrin formation. It circulates bound to von Willebrand factor, which protects it from rapid degradation. In hemophilia A, the lack of functional factor VIII leads to impaired thrombin production and inadequate fibrin formation, resulting in defective fibrin stabilization and failure of secondary hemostasis. This insufficiency ultimately causes the prolonged and recurrent bleeding characteristic of the disorder.

Treatment of hemophilia A involves targeted therapies such as prophylactic factor VIII infusions, subcutaneous emicizumab, and newer non-factor agents (marstacimab, concizumab, fitusiran), with desmopressin reserved for mild cases and immune tolerance therapy for those with inhibitors; in 2023, FDA approval of AAV-mediated gene therapy (valoctocogene roxaparvovec) provided a long-term option for adults with severe disease. Comprehensive care through hemophilia treatment centers includes patient education, home infusion training, physical therapy, pain management, and treatment of transfusion-related infections. Regular surveillance with inhibitor screening, bleeding assessments, musculoskeletal evaluation, and psychosocial support is essential, with closer monitoring for children and high-risk patients. Risk reduction strategies include avoiding circumcision until diagnosis is confirmed, minimizing trauma-prone activities, and cautious use of drugs affecting platelet function. Early genetic evaluation of at-risk relatives, particularly females before or during pregnancy, enables timely intervention, while affected women require careful monitoring during pregnancy and postpartum to prevent delayed bleeding.

 

"Hemophilia A- Pipeline Insight, 2025" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Hemophilia A pipeline landscape is provided which includes the disease overview and Hemophilia A treatment guidelines. The assessment part of the report embraces, in depth Hemophilia A commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Hemophilia A collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Hemophilia A R&D. The therapies under development are focused on novel approaches to treat/improve Hemophilia A.

 

Hemophilia A Emerging Drugs Chapters

This segment of the Hemophilia A report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, Preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

 

Hemophilia A Emerging Drugs

• FRSW117: Jiangsu Gensciences lnc.

FRSW117 is a next-generation, ultra-long-acting recombinant factor VIII therapy that combines Fc fusion and PEG technologies, enabling a convenient once-weekly dosing regimen. This prophylactic approach allows patients with hemophilia A to maintain a near-normal lifestyle and approach a “functional cure.” The product is protected by independent intellectual property rights, with core patents under substantive examination in more than 10 countries and regions, and multiple authorizations already granted. In September 2024, Gensciences received FDA orphan drug designation for its next-generation ultra-long-acting recombinant coagulation FVIII - Pegylated Recombinant Human Coagulation Factor Ⅷ-Fc Fusion Protein, intended for the treatment of hemophilia A. Currently, the drug is in Phase III stage of its clinical trial for Hemophilia A.

 

• Ex vivo hematopoietic stem cell (HSC) LV ET3 gene therapy: Expression Therapeutics, LLC

Ex vivo hematopoietic stem cell (HSC) lentiviral (LV) gene therapy utilizes a patient’s own autologous cells to restore clotting function. In this process, white blood cells are collected through apheresis, and hematopoietic stem and progenitor cells are isolated using CD34+ selection. These cells are then genetically modified with the proprietary LV-FVIII vector and reintroduced into the patient via a routine peripheral vein infusion. Once engrafted in the bone marrow, the modified CD34+ cells reestablish normal blood cell production while continuously generating functional factor VIII. This enables a long-term, self-sustaining supply of FVIII in the bloodstream, offering a potentially durable therapeutic solution for hemophilia A. Currently, the drug is in Phase I stage of its clinical trial for Hemophilia A.

 

• AMA006: Amarna Therapeutics

AMA006 is a gene replacement therapy designed for patients with hemophilia A (HEMA), a rare X-linked disorder caused by mutations in the gene encoding clotting factor VIII (FVIII). Similar to hemophilia B (HEMB), which results from factor IX (FIX) deficiency, HEMA leads to impaired blood clotting where even minor injuries can cause severe or life-threatening bleeding. While current management relies on lifelong factor replacement therapy, there is no definitive cure. Gene therapy using adeno-associated viral (AAV) vectors offers a promising alternative by delivering functional gene copies to the liver, enabling sustained clotting factor production. AMA006 employs the Nimvec™ vector to deliver the human FVIII gene, aiming to restore normal blood clotting and significantly reduce the treatment burden for patients. Currently, the drug is in Preclinical stage of its clinical trial for Hemophilia A.

 

• FVIII-CAART: Cabaletta Bio

FVIII-CAART is an investigational therapy being developed for patients with hemophilia A who develop inhibitors against factor VIII (FVIII), a serious complication that renders standard replacement therapy ineffective. Using a Chimeric AutoAntibody Receptor T cell (CAART) approach, it is designed to selectively target and eliminate FVIII-neutralizing alloantibodies and the B cells that produce them, thereby restoring the effectiveness of FVIII infusions and offering a potentially more durable and well-tolerated alternative to current immune tolerance strategies. Currently, the drug is in Discovery stage of its clinical trial for Hemophilia A.

 Further product details are provided in the report……..

 

Hemophilia A: Therapeutic Assessment

This segment of the report provides insights about the different Hemophilia A drugs segregated based on following parameters that define the scope of the report, such as:

 

Major  Players in Hemophilia A

• There are approx. 25+ key companies which are developing the therapies for Hemophilia A. The companies which have their Hemophilia A drug candidates in the most advanced stage, i.e. Phase III include, Jiangsu Gensciences lnc.

 

Phases

DelveInsight’s report covers around 30+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

 

Route of Administration

Hemophilia A pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

 

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

 

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

 

Hemophilia A: Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Hemophilia A therapeutic drugs key players involved in developing key drugs.

 

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Hemophilia A drugs.

 

Hemophilia A Report Insights

• Hemophilia A Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

 

Hemophilia A Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

 

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Hemophilia A drugs?
• How many Hemophilia A drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hemophilia A?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Hemophilia A therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Hemophilia A and their status?
• What are the key designations that have been granted to the emerging drugs?